The NIH Blueprint Neurotherapeutics Network is a program designed to discover and develop novel treatments for neurological diseases by providing support for small molecule drug discovery and development from hit-to-lead chemistry through Phase I testing. BPN14770 is the first compound under the program to reach a clinical trial. “We are pleased that BPN14770 has moved into a clinical study and we are eagerly awaiting the outcomes of the safety trial,” Amir Tamiz, PhD, program director at the NIH’s National Institute of Neurological Disorders and Stroke (NINDS), said in a press release.

BPN14770 is a phosphodiesterase 4D negative allosteric modulator (PDE4D-NAM), designed to both improve memory and slow AD progression. Research has shown that the brain neuronal enzyme phosphodiesterase sub-type 4D (PDE4D) modulates the formation of new synaptic connections between neurons, and plays a key role in learning and memory storage. Rolipram is a different type of PDE4 inhibitor that has been found to improve cognitive performance in AD mouse models, as well as in stroke and traumatic brain injury. However, rolipram is not clinically used due to its serious adverse side effects, making BPN14770 a potential treatment option for dementia and AD.

The Phase 1 clinical trial will investigate the pharmacokinetics and safety of BPN14770 in 48 healthy individuals. If a favorable safety profile is observed, the next stage will investigate its effects on long-term memory and other cognition aspects.

Many principal researchers may not have the resources or expertise to turn a newly discovered composite into treatment. The Blueprint Neurotherapeutics Network grants researchers access to drug discovery specialists who can assist them in both the discovery and development stages. “The goal of the Blueprint Neurotherapuetics Network is to rapidly move promising ideas for treating seemingly incurable neurological disorders from the lab bench to the bedside. The Network achieves this goal through collaborations between researchers, consultants and contractors. The consultants and contractors in the Neurotherapeutics Network provide their expertise to help investigators, many of whom may be new to drug development,” said Dr. Tamiz.

Future trials may assess the effects of the compound in patients with AD as well as in patients with mild cognitive impairment.

Disclaimer:

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